Aten previously raised $1 million from Srinivasan Namala, CEO of Hyderabad-based Porus Labs, who is now also a partner in the company. It also secured the Rs 50-lakh Biotechnology Ignition Grant instituted by the government-funded Biotechnology Industry Research Assistance Council.ET Bureau | February 03, 2017, 07:16 IST

To begin with, the duo decided to focus on Niemann-Pick Type C (NPC) disorder that’s also referred to as Childhood Alzheimer’s. In Pic: Aditya Kulkarni & Arun B Papaiah.BENGALURU: Aditya Kulkarni, a chemical biologist, became interested in rare diseases that afflict miniscule populations while pursuing his PhD at Purdue University. In 2014, after he returned to India, his college friend Arun Papaiah and he decided to establish Aten Porus Lifesciences to develop drugs to treat or manage these so-called orphan diseases.

“I thought that was one area where we can make an impact,” said Kulkarni, chief scientific officer at Aten Porus. “There are two main reasons as to why we picked orphan diseases—one, it a huge unmet medical need, and two, in the Indian perspective, the country has 70 million patients across 7,000 rare diseases.”

To begin with, the duo decided to focus on Niemann-Pick Type C (NPC) disorder that’s also referred to as Childhood Alzheimer’s. The disorder is characterised by the over-accumulation of cholesterol in the body in such a way that it causes severe damage to the brain, liver and spleen.

Globally, according to Kulkarni, fewer than 2,500 people are estimated to be afflicted with this disorder. Kulkarni went about formulating the drugs and began reaching out to experts at foreign universities for testing Aten’s formulations on animals.

The earliest breakthrough came from tests conducted by Maria Teresa Fiorenza, associate professor of applied biology in the Faculty of Medicine and Psychology at Sapienza University in Rome.

“It is a very niche area. If you take NPC, there are only 10-15 professors globally who are experts,” said Papaiah, chief executive of Aten Porus. “We asked (Fiorenza) to test our drug. We got very encouraging results. Not only could we show that the lifespan of mice could be doubled with our drug, but also that brain damage could be stopped.” The pair say their formulation is better than existing options as it allows for fewer doses to be administered and in easier ways.

Importantly, it can potentially result in a 30-50% reduction in cost. “Based on the strategy design of this drug and the encouraging results we have obtained up to now, I believe that it has the potential to represent a major breakthrough for the treatment of NPC disease and perhaps other lysosomal disorders,” said Fiorenza.

With the initial success, the duo wanted to test if the same thought process could be used to expand use of the drug for other cholesterol-related diseases or disorders. “If you look at the various diseases we want to tackle and distil them down, the underlying factor for each of these diseases is excess accumulation of cholesterol and lipids. What our drug could do is, the moment cholesterol is accumulated it is removed from the body,” said Kulkarni.

The plan is for Aten to partner with pharma companies that have marketing, production and distribution muscle. Aten Porus this week raised $1.4 million (about Rs 9.3 crore) for its subsidiary Oraxion Therapeutics from an undisclosed US-based pharmaceutical trading company.

Aten previously raised $1 million from Srinivasan Namala, CEO of Hyderabad-based Porus Labs, who is now also a partner in the company. It also secured the Rs 50-lakh Biotechnology Ignition Grant instituted by the government-funded Biotechnology Industry Research Assistance Council.

“Rare diseases have a fast-track approval with orphan designation and lot of incentives from regulatory bodies such as the USA-FDA and European-EMA,” said Namala. “The sheer number of (rare) diseases coupled with very few major pharma/biotechnology companies focusing on them offers a great opportunity for companies like (Aten).”